2025 Cure GM1 Impact Report
Check out what you helped us accomplish in 2025
All we’ve Achieved in 2025
THANK YOU. With your help, the Cure GM1 Foundation had a tremendous year fighting for greater awareness of, and research for, GM1 gangliosidosis. Each year, we publish an Impact Report so that our supporters can gain a glimpse of all they’ve helped us achieve with their time and financial investment. We are so grateful for your support.
Please download and share a PDF of the report at right. Or, check out a web-friendly summary of Cure GM1’s Impact Report below.
Research & Drug Development
ADVANCING ENZYME REPLACEMENT THERAPY FOR GM1
In early 2025, the Cure GM1 Foundation allocated an additional $400,000 to further the development of enzyme replacement therapy (ERT) as a potential treatment option for those living with GM1. This builds upon prior work towards enzyme replacement therapy. Cure GM1 anticipates considerable expansion of ERT efforts in 2026 and beyond.
GM1 Census
In October, Cure GM1 launched a new GM1 Census to help increase the data available for research regarding GM1. This study examines patient population data, including subtypes, diagnostic journeys, and associations between genetic variants and clinical symptoms. This registry expands on prior work on the Invitae Patient Insights Network. The census includes GM1 families in nearly 40 countries, from the United States to Egypt, Malta, Venezuela and beyond.
Clinical Research
In October, Cure GM1 launched a new GM1 Census to help increase the data available for research regarding GM1. This study examines patient population data, including subtypes, diagnostic journeys, and associations between genetic variants and clinical symptoms. This registry expands on prior work on the Invitae Patient Insights Network. The census includes GM1 families in nearly 40 countries, from the United States to Egypt, Malta, Venezuela and beyond.
World Symposium
In 2025, Cure GM1 presented two posters and abstracts at the WORLD Symposia Meeting in San Diego. One poster included the analysis of genotypes, signs, and systems from data collected on the AllStripes platform, which was acquired by Picnic Health for a cohort of 26 participants. In addition, Cure GM1 presented a poster on the formation of a GM1 data-sharing collaborative group, in an effort to support natural history data sharing.
Advocacy
GM1 Day
The 2025 GM1 Day led to 12 state and city proclamations. This included proclamations in
California, Illinois, Maryland, Massachusetts, Michigan, Missouri, Texas, Wisconsin, North Carolina; Carlsbad (CA), Milwaukee (WI), and Village of Rantoul (IL).
Community
Annual Community Conference
This year’s Cure GM1 Annual Community Conference was a hybrid event with international reach.
133 individuals registered
103 GM1 family Members REGISTERED
20 Countries
24 Speakers Presented
available in 50 languages
Notable Fundraisers
$30K MATCH FULFILLED AND EXCEEDED
Thanks to the generosity of Cure GM1’s supporters, this was a banner year for fundraising. The 2025 combined end-of-year/Giving Tuesday campaign was Cure GM1’s first to break six figures, hitting $102,057. This includes a fully maxed matching gift campaign of $30,000.
MULTIPLE VIDEO CAMPAIGNS FOR ERT & MORE
Thanks to everyone who donated and participated in the multiple fundraising campaigns throughout the year. Fundraisers included individual fundraisers, video campaigns, and the Do It for GM1 campaign.
Putting Hope in Motion in 2026
Looking ahead into 2026, the Cure GM1 Foundation plans to increase its support of enzyme replacement therapy and is committing an additional $1.4 million to further the development of this critically important treatment approach. And, as always, we’ll continue to push for awareness and increased support of GM1.
Consider a Gift to the Cure GM1 Foundation
Please know our ongoing growth and success in raising awareness and funding for GM1 gangliosidosis would not be possible without your support. Your help truly matters.